News brief­ing: Pfiz­er on track to ear­ly '22 piv­otal read­out on he­mo­phil­ia A; Co­di­ak scales back IPO ex­pec­ta­tions

Pfiz­er and Sang­amo have dosed their first pa­tient in the Phase III gene ther­a­py tri­al of giroc­toco­gene fitel­par­vovec (SB-525) for he­mo­phil­ia A. Re­searchers will be fol­low­ing the ex­pe­ri­ences of pa­tients for 12 months af­ter dose, check­ing on safe­ty, ef­fi­ca­cy and dura­bil­i­ty. The move comes on the heels of a ma­jor set­back at ri­val Bio­Marin, which re­ceived a re­jec­tion let­ter from the FDA, which de­mand­ed more proof of dura­bil­i­ty — a sore point among some of the an­a­lysts track­ing the pro­gram.

A spokesper­son for Pfiz­er tells End­points News to­day that they plan “a piv­otal read­out in ear­ly 2022 for giroc­toco­gene fitel­par­vovec.”

Bren­da Coop­er­stone, Pfiz­er’s chief de­vel­op­ment of­fi­cer, rare dis­ease, not­ed: “En­roll­ment in the lead-in study is pro­gress­ing well and re­cruit­ment is on track for Phase 3. Giv­en the Phase 1/2 study find­ings to date, we be­lieve that giroc­toco­gene fitel­par­vovec has the po­ten­tial to sus­tain fac­tor lev­els and re­duce an­nu­al bleed rates, sug­gest­ing this one-time gene ther­a­py could po­ten­tial­ly trans­form the stan­dard of care for el­i­gi­ble pa­tients world­wide.” — John Car­roll

Co­di­ak scales down IPO ex­pec­ta­tions to $83M

Amid a siz­zling hot biotech IPO en­vi­ron­ment where most com­pa­nies are bring­ing in more than they ini­tial­ly planned, Co­di­ak Bio­Sciences is tun­ing down ex­pec­ta­tions as it sets terms.

Hav­ing pen­ciled in $100 mil­lion in their IPO fil­ing, the ex­o­some spe­cial­ists now say they plan to raise $83 mil­lion by of­fer­ing 5.5 mil­lion shares at a price range of $14 to $16. That’s low­er than what they would have raised in 2019, when they launched but ul­ti­mate­ly pulled an at­tempt to go pub­lic due to un­fa­vor­able mar­ket con­di­tions.

At the mid­point of the pro­posed range, Co­di­ak would com­mand a ful­ly di­lut­ed mar­ket val­ue of $309 mil­lion, ac­cord­ing to Re­nais­sance Cap­i­tal.

A pi­o­neer in co-opt­ing ex­o­somes, the body’s nat­ur­al in­ter­cel­lu­lar com­mu­ni­ca­tion sys­tem, to de­liv­er ther­a­peu­tic pay­loads to spe­cif­ic ad­dress­es, Co­di­ak’s lead pro­gram is de­signed to ac­ti­vate the STING path­way to treat sol­id tu­mors. It ex­pects to start Phase I/II lat­er this year and reap pre­lim­i­nary da­ta by mid-2021. — Am­ber Tong

Pur­due re­port­ed­ly near­ing guilty plea in fed­er­al opi­oid probe

Pur­due Phar­ma is near­ing a plea deal where it would plead guilty to crim­i­nal charges re­lat­ed to its role in ag­gres­sive­ly mar­ket­ing high­ly ad­dic­tive opi­oids, ac­cord­ing to a Reuters re­port.

Run by the Sack­ler fam­i­ly, Pur­due would re­port­ed­ly face fines ex­ceed­ing $8 bil­lion should the agree­ment be reached. Those fines in­clude about $3.54 bil­lion in a crim­i­nal fine, a $2 bil­lion crim­i­nal for­fei­ture and a $2.8 bil­lion civ­il penal­ty, per Reuters

The agree­ment could be reached some­time with­in the next two weeks. Be­cause of Pur­due’s bank­rupt­cy pro­ceed­ings, the fines are un­like­ly to be paid out any­time soon giv­en that the com­pa­ny lacks the cash at present to re­pay all cred­i­tors.

Speak­ing to Reuters, a Jus­tice De­part­ment spokesper­son de­clined to com­ment but said that the re­port “con­tains in­ac­cu­ra­cies and is high­ly mis­lead­ing” with­out ex­plain­ing.

Pros­e­cu­tors across the coun­try, at the fed­er­al and state lev­els, have al­leged Pur­due made bil­lions of dol­lars in prof­its from its opi­oids and sent il­le­gal kick­backs to doc­tors and phar­ma­cies. Pur­due filed for bank­rupt­cy last year fol­low­ing sev­er­al law­suits and crim­i­nal cas­es over its role in fu­el­ing the opi­oid cri­sis. — Max Gel­man

Abeona forms spe­cial com­mit­tee to over­see lead­er­ship

Fol­low­ing a rough few years, Abeona Ther­a­peu­tics an­nounced the for­ma­tion of a spe­cial com­mit­tee of board mem­bers to over­see its ex­ec­u­tive lead­er­ship.

In ad­di­tion, the com­pa­ny has brought in Jef­feries to con­duct a re­view of strate­gic op­er­a­tions. The com­pa­ny said there’s no de­fined time­line for the re­view, and that it doesn’t in­tend to com­ment fur­ther un­less “a spe­cif­ic ini­tia­tive is ap­proved by the board of di­rec­tors, the re­view process is con­clud­ed, or it is oth­er­wise de­ter­mined that oth­er dis­clo­sure is ap­pro­pri­ate.”

Back in 2018, Abeona chief Carsten Thiel was oust­ed fol­low­ing ac­cu­sa­tions of un­spec­i­fied “per­son­al mis­con­duct.” And the fol­low­ing year, the FDA pumped the brakes on the biotech’s Phase III study of its po­ten­tial treat­ment for re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa. The FDA lift­ed its hold in De­cem­ber, al­low­ing Abeona to re­sume test­ing.

Last Oc­to­ber, Abeona’s $ABEO shares were at $2.38. On Tues­day, they closed at $1.22.

The spe­cial com­mit­tee will work “close­ly with the ex­ec­u­tive lead­er­ship team to de­vel­op the com­pa­ny’s strate­gic di­rec­tion and lead­er­ship plan,” Abeona an­nounced. — Nicole De­Feud­is 

Urovant and Sunovion strike mar­ket­ing deal for po­ten­tial over­ac­tive blad­der drug

Urovant has struck a 5-year deal with Sunovion Phar­ma­ceu­ti­cals to mar­ket its over­ac­tive blad­der drug vibegron in pri­ma­ry care in the US, should it get ap­proval in De­cem­ber.

Walt John­ston

The FDA ac­cept­ed Urovant’s NDA for vibegron in March, and set a PDU­FA date for Dec 26. Urovant in-li­censed the drug from Mer­ck with a $25 mil­lion up­front. Topline Phase III da­ta showed that it hit sta­tis­ti­cal sig­nif­i­cance for both co-pri­ma­ry end­points and all 7 sec­on­daries when com­pared against a place­bo.

Ac­cord­ing to the com­pa­ny, it’s al­so be­ing eval­u­at­ed for OAB in men with be­nign pro­sta­t­ic hy­per­pla­sia, and for ab­dom­i­nal pain as­so­ci­at­ed with ir­ri­ta­ble bow­el syn­drome.

“While Urovant sales rep­re­sen­ta­tives will be fo­cused on urol­o­gists, long-term care, and high-pre­scrib­ing PCPs, the Sunovion team will sig­nif­i­cant­ly broad­en our reach in­to the U.S. pri­ma­ry care com­mu­ni­ty,” Urovant’s se­nior com­mer­cial VP Walt John­ston said in a state­ment.

The new deal is in ad­di­tion to an ex­clu­sive 3-year agree­ment the com­pa­nies reached in June for whole­sale trade and re­tail dis­tri­b­u­tion, con­tract op­er­a­tions and se­lect ac­count man­age­ment for vibegron. — Nicole De­Feud­is 

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Paul Perreault, CSL Behring CEO

CSL CEO Paul Per­reault de­ter­mined to grow plas­ma col­lec­tion af­ter full-year sales dip

As the ink dries on CSL’s $11.7 billion Vifor buyout, the company posted a dip in profits, due in part to a drop in plasma donations amid the pandemic.

However, CEO Paul Perreault assured investors and analysts on the full-year call that the team has left “no stone unturned” when assessing options to grow plasma volumes. The chief executive also spelled out positive results for the company’s monoclonal antibody garadacimab in hereditary angioedema (HAE), though he isn’t revealing the exact numbers just yet.

Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Blaise Coleman, Endo International CEO

En­do files for Chap­ter 11 as it looks to fin­ish off its opi­oid lit­i­ga­tion

Irish drugmaker Endo International is entering into bankruptcy as it faces the weight of serious litigation related to its involvement in the opioid epidemic in the US.

The company has filed Chapter 11 proceedings in the US Bankruptcy Court for the Southern District of New York, with the company expected to file recognition proceedings in Canada, the UK and Australia. The company’s bankruptcy filing showed the company had assets and liabilities in the range of $1 billion to $10 billion.

FDA ap­proves one of the prici­est new treat­ments of all time — blue­bird's gene ther­a­py for be­ta tha­lassemia

The FDA on Wednesday approved the first gene therapy for a chronic condition — bluebird bio’s new Zynteglo (beti-cel) as a potentially curative treatment for those with transfusion-dependent thalassemia.

The thumbs up from the FDA follows a unanimous adcomm vote in June, with outside experts pointing to extraordinary efficacy, with 89% of subjects with TDT who received beti-cel having achieved transfusion independence.

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